4D-310 in Adults With Fabry Disease and Cardiac Involvement

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement

Inclusion Criteria:Adult males and femalesPathogenic GLA mutation consistent with Fabry DiseaseConfirmed diagnosis of classic or late-onset Fabry disease with cardiac involvementIndividuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollmentAgree to use highly effective contraceptionExclusion Criteria:Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGAeGFR <45 mL/min/1.73 m2Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysisHIV, active or chronic hepatitis B or C,Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic controlHistory of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)Contraindication to systemic corticosteroid therapy or immunosuppressive therapyChronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.Moderately severe to severe cardiovascular disease or uncontrolled hypertensionLeft ventricular ejection fraction of <45% on echocardiogram (ECHO)Currently receiving investigational drug, device or therapy or having ever received gene therapyHistory of infusion related response to ERT or any adverse reaction leading to ERT discontinuationHistory of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)Pregnant or breast-feeding